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Advocating for Americans’ Right to Try

By September 4, 2020No Comments

Amyotrophic lateral sclerosis (ALS) is a debilitating and terminal disease. Most patients live only two to five years after the onset of symptoms. Unfortunately, ALS has historically been underfunded for research and has few treatment options, none of which significantly delay progression or extend life. The New York Young Republican Club believes there has been a breakthrough treatment discovered, and we would like to see it made available to ALS patients as soon as possible.

AMX0035, produced by Amylyx, is an experimental combination of two existing drugs, sodium phenylbutyrate and taurursodiol, which can slow the progression of ALS. This is based on data from a recent Phase 2 clinical trial recently published in the New England Journal of Medicine. Traditionally, in order for this drug to get into the hands of patients with ALS, the FDA would require several steps and a Phase 3 clinical trial. This process typically takes anywhere from three to four years. Given the short life expectancy of ALS patients, most people currently fighting this disease will be dead before they could obtain this medication.

The New York Young Republican Club calls for the FDA to cut through red tape, meet with Amylyx, allow Amylyx to submit a New Drug Application, and approve said application swiftly on a preliminary basis for the treatment of ALS. After this, further testing on safety and effectiveness in the real-world can be done. Time is of the essence, and the longer it takes for the FDA to approve this drug, the more American lives will be lost to this terrible disease.

All Americans deserve the Right to Try.